Featured Blog Posts – October 2012 Archive (12)

Today's (October 31st) MD STARnet Webinar Postponed due to Hurricane

Dear Friends,

Due to the recent hurricane we are going to postpone tomorrow's MD STARnet webinar.  

We will be in touch regarding the new date. Sorry for the inconvenience. 


Ryan Fischer



Webinar: MD STARnet

Added by PPMD on October 30, 2012 at 1:00pm — No Comments

Partnering with the FDA to accelerate approvals- The question of risk tolerance in the Duchenne community


Friends, we have been actively pursuing a regulatory strategy and have had two meetings with the neurology division at the FDA. We are currently working on the 'to do' list for the community and are trying to understand barriers/obstacles to approval from the FDA side, as well as, from the sponsor side of the issue. We are working a smart, targeted call to action. We have learned through years…


Added by Pat Furlong on October 23, 2012 at 11:30pm — 6 Comments

Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy

New release from Prosensa:

£10m in milestone payments received from GlaxoSmithKline


Leiden, The Netherlands, 23 October 2012 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, has selected clinical candidates for two more compounds for the treatment of Duchenne muscular dystrophy (DMD) and has been granted…


Added by PPMD on October 23, 2012 at 12:35pm — 2 Comments

Facing reality

1997 may not seem so long ago, but at times it feels like half a lifetime to me. That was the year my family learned Matthew’s motor difficulties were something physical therapy could not correct and the first time we heard the word Duchenne. Everyone’s world changes after diagnosis of a devastating chronic illness and over the years my family and I experienced many…


Added by Brian Denger on October 22, 2012 at 8:00am — 3 Comments

Full 48-week Phase IIb data from eteplirsen study in Duchenne presented at World Muscle Society

Dr. Jerry Mendell, Director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital and principal investigator of the Phase IIb eteplirsen study, presented the full set of 48-week data from…


Added by PPMD on October 16, 2012 at 9:00am — No Comments

ImagingDMD: Making muscle biopsies a thing of the past

In the clinical trial world, there are some things that divide us these days- most specifically, what mutation type your son has. However, we are united in wanting better ways to look for the benefits of potential drugs; who isn’t hoping for a day when the muscle biopsy is a relic from the past? 

There are several dedicated research teams doing exciting work…


Added by Holly Peay on October 13, 2012 at 10:00am — No Comments

Management of End Stage Heart Failure: Ventricular Assist Devices (VAD)

In light of PPMD’s Cardiac Webinar Series, our Director of Care, Kathi Kinnett answers questions/concerns about Ventricular Assist Devices (VAD).


There has been recent discussion worldwide…


Added by Kathi Kinnett on October 11, 2012 at 2:30pm — No Comments

Better, Faster, Now!

These are the words that best describe PPMD’s research plan.

  1. We aim to put better drugs into development for clinical trials because we can’t afford the failures.
  2. We apply funding strategically to increase the speed of testing new drugs.
  3. We prioritize and fund the testing in Duchenne of drugs that are already approved for other purposes.

Everything we do goes through this filter to make sure that we are achieving one of these…


Added by Sharon Hesterlee on October 11, 2012 at 11:00am — 4 Comments

Positive Phase 1 trial results for SMT C1100 in Duchenne (Summit PLC)

Following closely on the heels of the good news about Sarepta’s exon 51 skipping strategy, PPMD is pleased to report that Summit…


Added by Sharon Hesterlee on October 10, 2012 at 10:30am — 13 Comments

Understanding Accelerated Approval

With the recent news of Sarepta's promising 48-week data, many have asked how accelerated approval may play into the FDA review of eteplirsen. Let's first define what accelerated approval means.


Because it…


Added by Ryan Fischer on October 5, 2012 at 2:00pm — 1 Comment

Promising News from Sarepta on Exon 51 Skipping Trial

The much-anticipated 48-week results on eteplirsen, a drug designed to skip exon 51, were presented today by Sarepta Therapeutics on a webinar and press release. On the webinar, Sarepta’s Chief Executive Chris Garabedian presented data about the dystrophin found in study participants’ muscle biopsies and the…


Added by Holly Peay on October 3, 2012 at 4:00pm — 5 Comments

The Global Genes Project

Jeans. Genes. Thursday night, I changed from jeans into a blue dress – as in blue jean blue. The recommended color for the R.A.R.E. Gala. The focus was genes, rare genetic disorders. While most of my days are focused on Duchenne, I am very aware that Duchenne is 1 of 7,000 rare…


Added by Pat Furlong on October 1, 2012 at 3:00pm — No Comments

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